Clinical Trials on Phenylketonuria (PKU) in New Zealand

Total 27 results

BioMarin Pharmaceutical

Recruiting

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Phenylketonuria (PKU)

United States
BioMarin Pharmaceutical

Active, not recruiting

AAV Gene Therapy Study for Subjects With PKU

Phenylketonuria (PKU)

United States, United Kingdom
BioMarin Pharmaceutical

Completed

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU (Prism-2)

Phenylketonuria (PKU)

United States
BioMarin Pharmaceutical

Recruiting

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) (PALace)

Phenylketonuria (PKU)

United States, Germany, Italy
BioMarin Pharmaceutical

Completed

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

Phenylketonuria (PKU)

United States
Homology Medicines, Inc

Enrolling by invitation

Long-Term Follow Up Study of Subjects Previously Administered HMI 102

Phenylketonuria | PAH Deficiency

United States
BioMarin Pharmaceutical

Completed

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

Phenylketonurias

United States
BioMarin Pharmaceutical

Completed

Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

Phenylketonurias

United States
BioMarin Pharmaceutical

Completed

A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

Phenylketonurias

United States
BioMarin Pharmaceutical

Completed

Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

Phenylketonuria

United States
Tulane University School of Medicine
BioMarin Pharmaceutical

Completed

Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders (IST)

Phenylketonuria | Behavior and Behavior Mechanisms | PAH Gene Expression

United States
BioMarin Pharmaceutical

Completed

A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks

Phenylketonuria

United States
BioMarin Pharmaceutical

Approved for marketing

Sapropterin Expanded Access Program

Phenylketonuria

United States
BioMarin Pharmaceutical

Completed

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

Phenylketonuria

United States
BioMarin Pharmaceutical

Completed

Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)

Phenylketonuria

United States
BioMarin Pharmaceutical

Completed

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

Phenylketonuria

United States
Synlogic

Completed

Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria (SynPheny-1)

Phenylketonuria

United States
BioMarin Pharmaceutical

Completed

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 (Prism301)

Phenylketonuria

United States
BioMarin Pharmaceutical

Completed

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry (PKUDOS)

Phenylketonuria | Hyperphenylalaninaemia

United States
BioMarin Pharmaceutical

Completed

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

Phenylketonuria

United States
BioMarin Pharmaceutical

Completed

A Prospective Clinical Study of Phenylketonuria (PKU)

Phenylketonurias

United States
Jnana Therapeutics

Recruiting

First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

Phenylketonuria

United States, Australia
Homology Medicines, Inc

Terminated

Gene Therapy Clinical Study in Adult PKU (pheNIX)

Phenylketonurias | PAH Deficiency

United States
PTC Therapeutics

Recruiting

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

Phenylketonuria

United States, Germany, Spain, Australia, Brazil, United Kingdom, Denmark, Mexico, Portugal, Turkey, Canada, Georgia, Italy, Netherlands, Japan
PTC Therapeutics

Completed

A Study of PTC923 in Participants With Phenylketonuria

Phenylketonuria

Spain, United States, Australia, Germany, United Kingdom, Brazil, Denmark, Canada, France, Georgia, Italy, Mexico, Netherlands, Portugal, Turkey
BioMarin Pharmaceutical

Completed

Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

Phenylketonuria

United States, Canada
BioMarin Pharmaceutical

Completed

Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency

Tetrahydrobiopterin Deficiencies | Hyperphenylalaninemia, Non-Phenylketonuric

Germany, United States

3

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